Leonard Lichtenfeld, MD: The TAPUR trial was started by ASCO, or The American Society of Clinical Oncology, and a lot of credit to them—the officers, the executives, and the members—for doing this. It was needed, it was necessary. We were out there in a space where everybody was saying, “I’m doing these genomic profiles, I’m getting the targets that might work, and I want to give my patient the medication. They don’t have any other option. Why shouldn’t I be able to give that medicine?” And of course, technically, there’s nothing preventing them from giving the medication, but the fact is that the cost is very high.

And unless you’re on a clinical trial, the payers and Medicare won’t pay for it. In fact, even today, if you find a patient with a mutation and you go to the insurance company and you say, “I want to use this medicine in this disease for this patient because they have a target,” the payer says, “We’re not going to pay for it, and we’re not only not going to pay it, we’re also not going to pay for any administration costs if we find out about it.” So, it’s pretty complex.

The TAPUR trial gives us the chance to take that technology, to take that science, and apply it to patients in real time, with everybody agreeing that they’re going to have access to the medicine, that they’re going to have payment for the treatments, and that the data are going to be available.

We live in an age where data should flow a lot more freely. That’s a whole other topic, I’m not going to get into that today, but the reality is that the data are not flowing as freely as they should. In this situation, take the information, look at it carefully, know what you’re looking for, and then see what we find out. The phrase that has been used is “basket trials.” So, there have been basket trials that have been done, and we’ve learned, for example, that some drugs that might work in one disease, even with a mutation—in a different disease, they don’t work. But even in the disease where it didn’t work, within a subset of patients, it worked. So, we have learned from doing this.

I think the real question is, how do we get more people involved? And not only just the health professionals, not just the FDA, but also, how do we get the community? How do we get consumers? How do we get caregivers to know that this is possible, and how do we get the doctors to engage? How do we get the community level physicians? Let us not forget that a lot of patients with cancer are treated in the community, and we have to make it reasonable for that doctor to participate—hopefully through TAPUR, hopefully through NCI-MATCH, and hopefully through other trials.

I also want to mention that it’s not just in these targeted therapies. On the West Coast, Dr. Laura Esserman, who’s at the University of California, San Francisco—I consider her a friend, and she’s a very forward-thinking individual and recognized as such—started adaptive trials, I-SPY, a number of years ago. She brought experts together in treatment of breast cancer, and without having to jump through all the hoops, they were able to change and move drugs in, move drugs out, and move things through the process faster.

Let’s not forget, it’s not just the FDA—well, maybe it is the FDA because it’s the clinical trial structure—but it’s also the institutional review board. It’s getting people to participate. It’s getting the doctors to participate, it’s getting the patients to participate, all of those pieces. If you have an ongoing trial mechanism, it makes life a lot easier and does accelerate the process. We’ve seen that happen now with other medications, and it’s certainly a trend that I think is heading in the right direction, but to make that happen, to make it really work, the pharmaceutical companies have to make the drug available and somebody has to pay for the administration of that drug.

The insurers have to step up to the plate—some do and some don’t—and not make it difficult, but say, “Yes, we understand that may not be a formal clinical trial, but it’s part of the learning process, and as long as there’s some organization to it, we’re willing to support that,” and do that research piece within the framework of traditional insurance. We’re going to have so many opportunities to advance medicine—to me, it’s incredible.

If we don’t figure out a way to move these medicines through the pipeline more quickly, we will all lose, because potentially useful medicines won’t become available to the people who need them as quickly as possible. We need to do better when it comes to getting patients into clinical trials. You can’t ask the question to somebody who has some expertise in this area without hearing that only 3% to 5%—maybe a little bit more, maybe a little less, depending on who gives the answer—of the patients who are eligible for clinical trials are put onto clinical trials.

Now there are a lot of reasons for that. Not every patient is treated at a major cancer center. Not every patient is treated at a large facility that has the resources to do clinical trials. There are a whole lot of people in this country who are in community settings—some in modest towns, some in small towns, some in rural America—who could also benefit from getting into clinical trials. So, we need to find mechanisms to identify those patients and get them in a trial at the place where they live.

Some will say, “Well, we don’t have oversight. We don’t need that.” I’ve actually been in a room with folks from a major-medical cancer center who said, “We don’t need those people from the community in our trials; we have more than enough at our place.” But even the major centers now have to band together. That’s a good thing, to have enough patients to try some of these drugs. There have been outreach efforts and they have been successful.

Lung-MAP is an example of where clinicians tried to go out and find these patients. They had an excellent response from community-based settings. It can be done. It needs to be done. If we’re going to take patients and put them through genomic testing, and we have a drug sitting over here that may match their somewhat unusual target, we need to get them together. And that means educating the patients and educating the professional staff.

We have a long way to go, by the way. It’s not just clinical trials, but also understanding genomics. The National Human Genome Research Institute had a conference recently about a program called GLEE (Genomic Literacy, Education, and Engagement). They talked about the need to educate communities; to educate health professionals; and to start, in elementary schools, teaching about genes and what they do, so we at least have an informed community. In the professional community, things are changing so quickly that the busy physician, the busy nurse, and the busy health professional do not have a whole lot of time to learn everything every day. It takes a lot of time and a lot of effort. So, we need to build the tools and the resources to make this thing work.

Again, there’s so much we don’t know yet and there’s so much we’re learning, and it’s changing, if not daily, quickly. If we don’t educate folks and get information to them on a point-of-service, real-time basis, then we will have failed to take advantage of the opportunity to improve, and maybe save, lives.

What Will Patients Gain From the TAPUR Study?

Leonard Lichtenfeld, MD, discusses the design of the TAPUR study, what patients will gain from it, and the approved targeted agents being studied.
Published Online: June 19, 2017
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Episode 1 Off-Label Communications: What It Means for Stakeholders
Episode 2 Considerations With Off-Label Communication
Episode 3 Evidence from Drug Manufacturers and Patient Access
Episode 4 Off-Label Communications to Inform Formulary Management
Episode 5 Improving the Drug Approval Process to Reduce Drug Costs
Episode 6 Drug Price Negotiation by CMS
Episode 7 Drug Price Arguments Have No Easy Answer
Episode 8 FDA Center of Excellence and the Drug Approval Process
Episode 9 Center of Excellence: Streamlining Drug Approvals
Episode 10 Empowering Stakeholders with Innovative Trial Designs
Episode 11 What Will Patients Gain From the TAPUR Study?
Episode 12 Engaging Patients in Oncology Trials Is a Team Effort